News & Stories
See the latest news about CMT drug development and read stories from the CMT community that highlight why we must deliver treatments and cures during our lifetime.
CMT Research Foundation Invests in XtRNA Bio to Develop Gene Therapy for CMT2D
CMT Research Foundation has invested in a research project with XtRNA Bio aimed to develop a new viral gene therapy for Charcot-Marie-Tooth disease type 2D. How GARS1 Mutations Drive CMT2D CMT2D is caused by mutations in the GARS1 gene, which encodes glycyl-tRNA...
A Father Who Defies ‘Impossible’: Joao Cardoso’s Journey with CMT
Joao Cardoso, Brittany, France, Living with CMT Joao Cardoso never imagined that he would be riding through the town of Brittany, France, in a wheelchair, while his eight-year-old son, Edgar, pedals his bike alongside him. Because of Joao’s Charcot-Marie-Tooth...
Applied Therapeutics Presents Findings From Phase 2/3 Clinical Trial of Govorestat in CMT-SORD
Applied Therapeutics recently presented findings from their INSPIRE Phase 2/3 clinical trial of govorestat (AT-007) for the treatment of Sorbitol Dehydrogenase Deficiency, a subtype of Charcot-Marie-Tooth disease, at the Peripheral Nerve Society’s 2025 Annual Meeting...
CMT Research Foundation Invests in Asha Therapeutics to Test Novel Drug for CMT2A
CMT Research Foundation has invested in a research project with Asha Therapeutics to investigate the therapeutic potential of inhibiting SARM1 (Sterile alpha and TIR motif-containing protein 1) using their novel drug, ASHA-624, for the treatment of Charcot-Marie-Tooth...
Vanderbilt University Researchers Identify Promising Compounds Targeting PMP22 Protein Expression, Paving the Way for New Treatments for CMT1A, CMT1E and HNPP
Researchers at Vanderbilt University have made significant progress in the development of small molecules aimed at treating Charcot-Marie-Tooth disease types caused by mutations in the PMP22 gene, which include CMT1A, CMT1E and hereditary neuropathy with liability to...
Because of Luca: A Family’s Fight Against CMT
Tara, Luca, Liam and Lane, Louisiana, Living With CMT When Luca Haik was just four years old, his mother, Tara, sensed something wasn’t right. “Luca came home from school every day crying in pain, rushing to take off his socks and shoes saying that his feet ‘felt...
82VS Progressing Ahead of Schedule in CMTRF-Funded Project to Improve Oligonucleotide Drug Delivery for CMT
Since launching their collaboration with CMT Research Foundation in November 2024, researchers at 82VS, the venture studio of Alloy Therapeutics, have made rapid progress and are ahead of schedule testing their novel technology focused on improving RNA therapeutic...
More Than Just a Rare Disease: Jodi Balog’s Journey With CMT
Jodi Balog, Pennsylvania, Living With CMT Jodi Balog has lived with Charcot-Marie-Tooth disease for more than 30 years. Diagnosed at the age of 13, CMT has become all she’s ever known. While CMT is classified as a rare disease, for Jodi — and millions of others — it’s...
NMD Pharma Announces FDA Orphan Drug Designation for NMD670
NMD Pharma has announced that they have been granted orphan drug designation by the Food and Drug Administration for NMD670, their novel, oral, small molecule inhibitor of the skeletal muscle-specific chloride ion channel ClC-1, for the treatment of...
NMD Pharma Publishes Findings Supporting the Role of ClC-1 Inhibition in Charcot-Marie-Tooth Disease
NMD Pharma announced that they have published new clinical and preclinical data that provides evidence that targeting neuromuscular junction deficits could potentially improve muscle function in CMT patients. The peer-reviewed paper titled “Neuromuscular Transmission...